Amsterdam Molecular Therapeutics Provides Operations Update & Strategic Review of Pipeline‏

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today provided an update on operations and disclosed the outcome of a strategic review of its gene therapy product pipeline conducted by management and AMT’s Board of Directors that enables the company to remain at the forefront of gene therapy development. The CHMP’s recent opinion, while not approving Glybera®, did not identify any safety risks with AMT’s adeno-associated virus vector technology. This will allow AMT to leverage its expertise and strength in building other portfolio products. The selection of specific pipeline products is aimed at securing partnering agreements that will provide solid financial foundations to take the company forward. Existing shareholders continue to see promise in the technology and are in discussions with AMT to provide additional equity.

-Pipeline
AMT will focus development efforts and financial resources on three gene therapy programs: hemophilia B, GDNF and acute intermittent porphyria. The Company will continue participating in the SanfilippoB program, which is fully funded by a consortium led by Institut Pasteur. Further investment in and development of Glybera and the Duchenne’s muscular dystrophy program has been suspended.

The company intends to support the development of its product pipeline through partnering agreements. Negotiations are being conducted for two programs.

-Glybera®
Following the negative opinion provided by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for Glybera (alipogene tiparvovec) as a treatment of lipoprotein lipase deficiency (LPLD), AMT has suspended the program. Consequently, AMT does not expect that Glybera will reach European patients. AMT decided that further investment in Glybera, in light of requirements necessary for approval and with the current financial resources available to the company, is no longer tenable.

-Background to Regulatory Process
In 2009, AMT filed Glybera for European marketing authorization “under exceptional circumstances”. In Europe, this submission process is intended to allow drugs for extremely rare diseases to be approved, realizing that the clinical development package will never contain all the information that would be required for a more common condition.

The dossier was examined by the Committee for Advanced Therapies (CAT) and the CHMP, and in June 2011 both voted against approval of Glybera. AMT appealed the decision based on its interactions with representatives from the CHMP and two new CHMP Rapporteurs were appointed to lead the review process. During the appeal, both rapporteurs, an external scientific advisory group (SAG) consisting of European experts in the areas of gene therapy, lipid metabolism and pancreatitis, was appointed to review and analyze the data and to advise the EMA on its conclusions. The SAG concluded that Glybera should be approved under exceptional circumstances. Subsequently, the CAT performed its own analysis and also voted by a large majority in favor of Glybera marketing authorization. The advice from the SAG, the CAT and both the CHMP Rapporteurs, was provided to the CHMP. However, the CHMP did not change its previous opinion and again voted with the narrowest majority against Glybera marketing authorization. The Company has not yet been informed of the reasons for the CHMP decision.

-Operations
Following a careful review of operations and resources needed for development of its remaining pipeline products, AMT management will work in close consultation with the works council to assess the impact of the review on the company’s workforce. Management and board believe that for AMT to remain a viable business it needs to reduce the headcount by 50% to 45 full-time employees.

-Financials
The measures announced today, including the refocusing of the product pipeline and reduction in headcount, will reduce the Company’s burn rate to approximately EUR500-600,000 per month. At June 30, 2011 AMT had cash and cash equivalents of EUR 9.1 million.

“The loss of jobs and the knowledge that LPLD patients in Europe will not be able to have access to an effective treatment is a huge regret for the management and board of AMT,” explained Jörn Aldag, CEO of AMT. “The decision to restructure the company has not been taken lightly but it now means there is a future for AMT. With the support of our investors and the anticipated closing of partnerships, we believe we can manage the cash position to sustain the company for at least the next 2 years.”